- After a three-month delay on its original user fee action date, the Food and Drug Administration has now issued a complete response letter for Eli Lilly & Co and Incyte's long-heralded oral rheumatoid arthritis drug baricitinib.
- The FDA's complete response letter (CRL) stated that it was unable to approve the application in its current form, and that it required additional data are necessary to determine the most appropriate doses and to further characterize safety concerns across treatment arms.
- Both companies disagree with the FDA's conclusions over the once-daily oral JAK1/2 inhibitor, and resubmission will be based on further discussions with the agency.
"The letter indicates that the FDA is unable to approve the application in its current form. Specifically, the FDA indicated that additional clinical data are needed to determine the most appropriate doses. The FDA also stated that additional data are necessary to further characterize safety concerns across treatment arms," said the company's about the CRL.
This was a huge hit for both Lilly and Incyte. For the big pharma, the company has been relying on the blockbuster potential of the RA drug to prove out its late-stage pipeline. For Incyte, which discovered the compound, the setback could be a financial one. Incyte said in a statement this weekend that it will now re-evaluate its previously announced guidance.
Evercore ISI analyst Umer Raffat highlighted in a note to investors that the FDA's request for more data was a bit startling and that it raises further questions about the safety of the 4mg dose, which had been presumed to be the more "appropriate dose."
As Raffat concluded, there needs to be more information from the companies to be able to understand the details of the feedback from the FDA, but he believes that it could perhaps be a focus on whether 2mg is the more appropriate dose.
Meanwhile, Jefferies analysts wondered in their own note to clients whether the uncertainties about malignancies or the maintenance dose characterization could be behind the FDA's caution. They suggest that this could push out the timeline a further year, have an impact on milestones and royalties, and may even affect EU income as well.
Last December, the European Medicines Agency's Committee for Medicinal Products for Human Use recommended the approval of Lilly and Incyte's baricitinib, followed by an approval under the name Olumiant in February as both a 2mg and 4mg dose. It's of note, though, that while the EMA approved both doses, it concluded the 4mg dose was the more appropriate.
While the RA space is a crowded one with lots of generic treatments and several other newer options, the rejection sets up competitors Sanofi and Regeneron to bring their own new RA treatment sarilumab to market—the drug was previously rejected due to manufacturing problems.